Fort Worth

Rainwaters assemble a team to find a treatment for a rare, deadly disease

Researchers in the Tau Consortium gathered Monday at the Fort Worth Club. The consortium is working on a treatment for progressive supranuclear palsy, a degenerative brain disease.
Researchers in the Tau Consortium gathered Monday at the Fort Worth Club. The consortium is working on a treatment for progressive supranuclear palsy, a degenerative brain disease. Star-Telegram

Shortly after Richard Rainwater was diagnosed with a degenerative brain disease in 2009, the billionaire Fort Worth investor gathered with family members and a team of doctors in New York to learn more about progressive supranuclear palsy.

The facts were sobering. There were no cures and no proven treatments for PSP, a rare and fatal disorder that only affects about six in every 100,000 people. Initially, patients typically have problems with their balance or show personality changes. Eventually they lose the ability to speak and swallow.

But Rainwater, who made a name for himself in the business world as a bold dealmaker with an eye for attracting top talent, wasn’t about to give in.

“I remember, during a break, Richard looked at me and said, ‘If money can beat this thing, we’re gonna beat it,’” said Walter Rainwater, Richard’s brother.

Over the last five years, Rainwater has poured more than $50 million into a research effort known as the Tau Consortium, which coordinates the work of about 35 leading doctors and scientists in the United States and Europe with the express goal of developing treatments for PSP. The project is named after the tau protein, which is believed to malfunction and be at the core of various brain diseases including Alzheimer’s.

This week, the consortium’s researchers gathered at the Fort Worth Club for a semi-annual meeting, where they shared results and updated each other on their work. The group’s funding has come from both Rainwater and his Rainwater Charitable Foundation, which developed the consortium and intends to provide $12 million for research in 2015.

The effort has produced quick results. Already, new treatments are being given to PSP patients as a result of the consortium’s work, and clinical trials are expected to begin this year on other treatments, designed to break up tau tangles in the brain and clear it from the body.

Dr. Bruce Miller, director of the Memory and Aging Center at the University of California at San Francisco (UCSF), who acts as scientific director for the consortium, said its work has been “transforming” in his field.

“This is one of the biggest efforts, I think, in the history of science to treat neurodegenerative diseases,” he said. “This has been accelerated science, science on steroids.”

While PSP afflicts only a small number of people, Miller said, the consortium believes its work could eventually help millions suffering from tau-related disorders, including Alzheimer’s patients and professional football players who develop chronic traumatic encephalopathy, or CTE.

For Todd Rainwater, Richard’s son, who is working with the consortium through the family foundation along with his brother, Matt, the effort provides a channel to work each day to help their father. But he knows time is not on their side.

“When you have a relative come down with this disease, most people don’t have any control over that,” he said.

“I know that it’s a long shot, but having a little sense of control really makes me feel very privileged,” he said. “And if we can’t find something for him, then finding something for the next generation and the next generation, that’s exciting.”

The family is guarded about revealing too much about Richard Rainwater’s condition. In a statement, they said that “mobility and communication are gradually becoming more of a challenge” but that the 70-year-old investor “still visits the office for a few hours nearly every day and enjoys socializing with friends and family.”

Consortium leaders declined to say whether Rainwater would be involved in the upcoming clinical trials, but the purpose of their work is clear.

“Ultimately, the goal is a therapy,” said Adam Boxer, another UCSF scientist who is leading the clinical trials effort. “We need to get a therapy for Richard and other patients as quickly as possible.”

Building a brain trust

Todd Rainwater said the family foundation, which also supports a number of educational causes, has been “very impressed” by the consortium’s work.

In less than five years, researchers have identified certain genes as risk factors for PSP, developed mouse and human stem cell models for studying the disease, developed lead compounds for treatment and built a clinical trial network.

“It often takes decades to develop a new treatment, but we believe that we’re already on the threshold of a major breakthrough,” said Patrick Brannelly, the consortium’s program director.

The consortium’s leaders credit the progress to its collaborative structure, a trait borrowed from Richard Rainwater’s business philosophy.

A Fort Worth native, Richard Rainwater was tapped by Sid Bass, a classmate at Stanford University’s business school, to manage the Fort Worth family’s money. He famously led a Bass investment in The Walt Disney Co., which turned itself around under the guidance of Michael Eisner and greatly expanded the family’s wealth.

After going out on his own, Rainwater joined other investors including George W. Bush as owners of the Texas Rangers baseball team, and helped to launch companies including Columbia/HCA Healthcare, Crescent Real Estate and Pioneer Natural Resources. Last year, Forbes magazine estimated Rainwater’s net worth at $2.7 billion.

As with his businesses, Rainwater sought out the best minds for the consortium and has encouraged them to work together rather than conduct research in academic silos that might take years to get published. At the group’s semi-annual meetings, the researchers sit around tables and discuss findings that they might never before have been comfortable sharing with each other, Brannelly said.

“The whole idea behind the consortium is to bring these people together in a way that accelerates the process,” he said.

Dr. Stanley Prusiner, who won the Nobel Prize in medicine in 1997 for discovering the cause of mad cow disease and its human equivalent, is working on drug development at his lab at UCSF. He said the consortium has been “visionary” in coordinating research and providing resources, such as an advanced microscope he is using to analyze cells.

Marc Diamond, who joined UT Southwestern in Dallas last fall as director of its new Center for Alzheimer’s and Neurodegenerative Diseases, said being part of the consortium has allowed him to meet colleagues from around the world and exchange ideas. “This is definitely unique,” he said.

Providing hope

For Darlene Boudreaux, the Tau Consortium represents hope for families touched by devastating diseases.

She recalled that when her husband, Dick Ryan, was diagnosed with PSP at the age of 63 in December 2003, there were no treatments available. He eventually developed motor skill problems similar to patients with Lou Gehrig’s disease, and died in November 2010.

“This disease was so incredibly hopeless because nobody was focusing on cures or treatments for it,” said Boudreaux, who is executive director of the Tech Fort Worth business incubator. “As a family member, it’s the hopelessness that they’re combating.”

Susan Dickinson, executive director of the Association for Frontotemporal Degeneration, a patient advocacy group based in Radnor, Pa., said the progress that’s being made is “very exciting.” Typically, patients have a life span of 7 to 8 years after being diagnosed, though with good care some can live for more than a decade.

“There is an extreme urgency to this work,” she said. “There’s never going to be a lot of money invested in a rare disease, which is why when one family comes along with the means they have and are willing to fund it to this extent, they are definitely having an impact.”

Miller, who has spent more than 30 years working on neurological diseases, said the consortium has been “a dream come true” and he credits the Rainwater family for its progress.

“This is an unusual family for me in terms of philanthropy,” he said. “They’re fun, they’re unpretentious, they’re very direct … their selflessness is something we are trying to emulate.”

Steve Kaskovich, 817-390-7773

Twitter: @stevekasko